CR20220567A - Compuestos y métodos para modular el empalme - Google Patents

Compuestos y métodos para modular el empalme

Info

Publication number
CR20220567A
CR20220567A CR20220567A CR20220567A CR20220567A CR 20220567 A CR20220567 A CR 20220567A CR 20220567 A CR20220567 A CR 20220567A CR 20220567 A CR20220567 A CR 20220567A CR 20220567 A CR20220567 A CR 20220567A
Authority
CR
Costa Rica
Prior art keywords
methods
compounds
splicing
modulating splicing
modulating
Prior art date
Application number
CR20220567A
Other languages
English (en)
Inventor
Frederic Vaillancourt
Peter Smith
Anant A Agrawal
Michael W Seiler
Serge Leger
Sudeep Prajapati
Dominic Reynolds
Original Assignee
Remix Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Remix Therapeutics Inc filed Critical Remix Therapeutics Inc
Publication of CR20220567A publication Critical patent/CR20220567A/es

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D495/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms
    • C07D495/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
    • C07D495/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D513/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups C07D463/00, C07D477/00 or C07D499/00 - C07D507/00
    • C07D513/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups C07D463/00, C07D477/00 or C07D499/00 - C07D507/00 in which the condensed system contains two hetero rings
    • C07D513/04—Ortho-condensed systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D519/00—Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Replacement Of Web Rolls (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Oxygen Or Sulfur (AREA)
  • Saccharide Compounds (AREA)

Abstract

La presente divulgación presenta compuestos y composiciones relacionadas que, entre otras cosas, modulan el empalme de ácidos nucleicos, p. ej., el empalme de un pre-ARNm, así como métodos de uso de estos
CR20220567A 2020-04-08 2021-04-08 Compuestos y métodos para modular el empalme CR20220567A (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063007327P 2020-04-08 2020-04-08
US202063043920P 2020-06-25 2020-06-25
US202063072873P 2020-08-31 2020-08-31
US202063126493P 2020-12-16 2020-12-16
PCT/US2021/026477 WO2021207550A1 (en) 2020-04-08 2021-04-08 Compounds and methods for modulating splicing

Publications (1)

Publication Number Publication Date
CR20220567A true CR20220567A (es) 2023-01-13

Family

ID=75747105

Family Applications (1)

Application Number Title Priority Date Filing Date
CR20220567A CR20220567A (es) 2020-04-08 2021-04-08 Compuestos y métodos para modular el empalme

Country Status (13)

Country Link
EP (2) EP4132936A1 (es)
JP (2) JP2023520925A (es)
KR (2) KR20230004575A (es)
CN (2) CN116134036A (es)
AU (2) AU2021253571A1 (es)
BR (2) BR112022020418A2 (es)
CA (2) CA3175193A1 (es)
CL (1) CL2022002779A1 (es)
CO (1) CO2022015926A2 (es)
CR (1) CR20220567A (es)
IL (1) IL297149A (es)
MX (2) MX2022012676A (es)
WO (2) WO2021207530A1 (es)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111499615B (zh) 2017-08-04 2024-02-02 斯基霍克疗法公司 用于调节剪接的方法和组合物
US11806346B2 (en) 2020-05-13 2023-11-07 Chdi Foundation, Inc. HTT modulators for treating Huntington's disease
IL305203A (en) * 2021-03-17 2023-10-01 Hoffmann La Roche New histories of thiazolopyrimidines
EP4308577A1 (en) * 2021-03-17 2024-01-24 F. Hoffmann-La Roche AG New thiadiazolopyrimidone derivatives
WO2023064879A1 (en) * 2021-10-13 2023-04-20 Remix Therapeutics Inc. Compounds and methods for modulating nucleic acid splicing
WO2023097007A1 (en) * 2021-11-24 2023-06-01 Remix Therapeutics Inc. Compounds and methods for modulating splicing

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7745447B2 (en) * 2005-10-26 2010-06-29 Bristol-Myers Squibb Company Substituted thieno[3,2-D]pyrimidines as non-basic melanin concentrating hormone receptor-1 antagonists
US8173661B2 (en) * 2006-11-08 2012-05-08 The Rockefeller University Alpha-IIB-beta-3 inhibitors and uses thereof
CA2805724A1 (en) * 2010-07-16 2012-01-19 Rockefeller University Oxadiazolo[3,2-a]pyrimidines and thiadiazolo[3,2-a]pyrimidines
SG10201609188WA (en) 2012-02-10 2016-12-29 Ptc Therapeutics Inc Compounds for treating spinal muscular atrophy
MY174339A (en) 2012-08-13 2020-04-09 Novartis Ag 1,4-disubstituted pyridazine analogs and methods for treating smn-deficiency-related conditions
JP6884102B2 (ja) 2015-02-09 2021-06-09 エフ・ホフマン−ラ・ロシュ・アクチェンゲゼルシャフト がんの治療のための化合物
EP3310169B1 (en) 2015-05-30 2023-05-17 PTC Therapeutics, Inc. Methods for modulating rna splicing
WO2017100726A1 (en) 2015-12-10 2017-06-15 Ptc Therapeutics, Inc. Methods for treatng huntington's disease
CA3037642A1 (en) * 2016-09-23 2018-03-29 Bayer Aktiengesellschaft N 3 -cyclically substituted thienouraciles and use thereof
CA3043755A1 (en) 2016-11-28 2018-05-31 Ptc Therapeutics, Inc. Methods for modulating rna splicing
JP2020523365A (ja) 2017-06-14 2020-08-06 ピーティーシー セラピューティクス，インコーポレーテッド Ｒｎａスプライシングを改変する方法
WO2018237084A1 (en) * 2017-06-21 2018-12-27 SHY Therapeutics LLC COMPOUNDS INTERACTING WITH RAS SUPERFAMIL FOR USE IN THE TREATMENT OF CANCERS, INFLAMMATORY DISEASES, RASOPATHIES AND FIBROTIC DISEASES
CN111499615B (zh) 2017-08-04 2024-02-02 斯基霍克疗法公司 用于调节剪接的方法和组合物
WO2019060917A2 (en) 2017-09-25 2019-03-28 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR SCREENING AND IDENTIFYING SPREADING MODULATORS
KR20200142039A (ko) 2018-04-10 2020-12-21 스카이호크 테라퓨틱스, 인코포레이티드 암 치료용 화합물
EA202190116A1 (ru) 2018-06-27 2021-08-30 Реборна Биосайенсиз, Инк. Агент для профилактики или лечения спинальной мышечной атрофии

Also Published As

Publication number Publication date
WO2021207550A1 (en) 2021-10-14
AU2021253571A1 (en) 2022-11-10
CA3175193A1 (en) 2021-10-14
MX2022012677A (es) 2023-01-11
JP2023520925A (ja) 2023-05-22
CO2022015926A2 (es) 2022-11-29
BR112022020418A2 (pt) 2023-05-02
CN116096725A (zh) 2023-05-09
JP2023520924A (ja) 2023-05-22
BR112022020337A2 (pt) 2022-12-13
WO2021207530A8 (en) 2021-12-02
CA3175205A1 (en) 2021-10-14
EP4132935A1 (en) 2023-02-15
MX2022012676A (es) 2023-01-11
WO2021207530A1 (en) 2021-10-14
EP4132936A1 (en) 2023-02-15
CL2022002779A1 (es) 2023-04-28
KR20230005210A (ko) 2023-01-09
KR20230004575A (ko) 2023-01-06
AU2021251220A1 (en) 2022-11-03
IL297149A (en) 2022-12-01
CN116134036A (zh) 2023-05-16

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